BioMarin Pharmaceutical Inc., a leading global biotechnology entity, unveiled that its drug, VOXZOGO (vosoritide), has received the green light from the U.S. Food and Drug Administration (FDA) to enhance linear growth in pediatric patients of all ages with open epiphyses (growth plates) suffering from achondroplasia.

Key Highlights from the Approval

•  Previously, the drug’s indication was limited to children aged five and above.
•  The FDA’s accelerated approval was grounded in a rise in annualized growth velocity.

“We are pleased that VOXZOGO is now available for children of all ages with achondroplasia,” commented Hank Fuchs at BioMarin.

Clinical Backing and Global Presence of VOXZOGO

The efficacy of VOXZOGO was backed by a Phase 2 clinical trial for children below 5, and its safety profile resembled that in older children. Data revealed notable average height improvements.

Dr. William Wilcox noted the significance of this expansion, highlighting that starting treatments earlier could offer a prolonged treatment window. Since VOXZOGO’s introduction in 2021, BioMarin has witnessed robust global patient demand, ensuring increased commitments for 2024 to cater to the rising need.

VOXZOGO’s global approval includes Europe, Japan, Brazil, and Australia, with diverse age criteria.

Future Outlook and Additional Details

The drug has also bagged orphan drug designation from the FDA for hypochondroplasia, another genetic condition influencing bone growth. BioMarin is geared up to commence a pivotal development program for hypochondroplasia later in the year.

At its core, VOXZOGO addresses a bone growth issue in children with achondroplasia by positively regulating the signaling pathway.

  achondroplasia, BioMarin Pharmaceutical, Dr. William Wilcox, Hank Fuchs, open epiphyses, US FDA, USA, VOXZOGO